For Rare Disease Patients, a Pathway to Hundreds of New Therapies March 21, 2017 Share this page Rare disease patients use drugs off-label based on limited data because they have no better options available Related Articles A Mid-Year State Policy Recap July 22, 2026 The EveryLife Foundation Submits Comments to Support Patient-Focused Drug Development and Medical Innovation July 01, 2026 FDA Rare Disease Roundtable – June 3, 2026 June 04, 2026 See All News